December 19, 2013 8:30am EST
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MIAMI--(BUSINESS WIRE)--
OPKO Health, Inc. (NYSE: OPK), has completed patient recruitment in the
second phase 3 trial of Rayaldy TM to treat
patients with secondary hyperparathyroidism (SHPT), stage 3 or 4 chronic
kidney disease (CKD) and vitamin D insufficiency. This trial is the
second of two identical randomized, double-blind, placebo-controlled,
multi-site studies intended to establish the safety and efficacy of Rayaldy TM as
a new treatment for SHPT in the targeted population. The endpoints of
both studies, which are being conducted in parallel, include vitamin D
status and changes in serum calcium, serum phosphorus and plasma intact
parathyroid hormone (PTH).
Each of the two pivotal phase 3 trials involves approximately 210
patients recruited at approximately 40 sites in the United States
(U.S.). Patients are being stratified by CKD stage and randomized in a
2:1 fashion to receive six months of treatment with either Rayaldy TM or
placebo. Top-line data from both trials are expected in mid-2014.
The two pivotal trials are being followed by an open-label extension
study in which patients are treated, at their election, for an
additional 6 months with Rayaldy TM . More than
50% of the required patients have enrolled in this open-label study and
some have already completed treatment.
About Rayaldy TM
Rayaldy TM is a first-in-class oral vitamin D
prohormone treatment being developed for SHPT in stage 3 and 4 CKD
patients with vitamin D insufficiency. It has a proprietary
modified-release formulation designed to gradually and reliably raise
serum total 25-hydroxyvitamin D (prohormone) concentrations to targeted
levels (at least 30 ng/mL) while avoiding upregulation of CYP24, a
cytochrome P-450 enzyme that reduces the PTH lowering potency of current
vitamin D supplements. Activation of calcifediol, the active ingredient
in Rayaldy TM , by the kidney is tightly regulated,
preventing excessive elevation of serum calcium and related side effects
which encumber current vitamin D hormone therapies and promote vascular
and renal calcification. Once approved, Rayaldy TM is
expected to address the approximately 4 million CKD stage 3 and 4
patients in the U.S. and many more, elsewhere, with SHPT and vitamin D
insufficiency.
About Chronic Kidney Disease
CKD is a condition characterized by a progressive decline in kidney
function. The kidney is normally responsible for excreting waste and
excess water from the body, and for regulating various hormones. CKD is
classified in five different stages — mild (stage 1) to severe (stage 5)
disease — as measured by the kidney's glomerular filtration rate.
According to the National Kidney Foundation, CKD afflicts over 26
million people in the U.S., including more than eight million patients
with moderate (stages 3 and 4) and severe (stage 5) forms of CKD. In
stage 5 CKD, kidney function is minimal to absent and patients require
regular dialysis or a kidney transplant for survival.
About Vitamin D Insufficiency
Vitamin D insufficiency is a condition in which the body has low vitamin
D stores, characterized by inadequate blood levels of vitamin D
prohormone, known as 25-hydroxyvitamin D. An estimated 70-90% of CKD
patients have vitamin D insufficiency, which can lead to SHPT and
resultant debilitating bone diseases.
About Secondary Hyperparathyroidism (SHPT)
SHPT is a condition commonly associated with CKD in which the
parathyroid glands secrete excessive amounts of PTH. SHPT arises as a
result of vitamin D insufficiency or impaired kidney function that
prevents sufficient production of vitamin D hormone to properly regulate
calcium and phosphorus metabolism, and PTH secretion. Prolonged
elevation of blood PTH causes excessive calcium and phosphorus to be
released from bone, leading to elevated serum calcium and phosphorus,
softening of the bones (osteomalacia) and calcification of vascular and
renal tissues. SHPT affects 40-60% of patients with moderate CKD and
approximately 90% of patients with severe CKD.
About OPKO Health, Inc.
OPKO is a multi-national biopharmaceutical and diagnostics company that
seeks to establish industry-leading positions in large and rapidly
growing medical markets by leveraging our discovery, development and
commercialization expertise and our novel and proprietary technologies.
For more information, visit our website at http://www.opko.com .
This press release contains "forward-looking statements," as that
term is defined under the Private Securities Litigation Reform Act of
1995 (PSLRA), regarding product development efforts and other
non-historical facts about our expectations, beliefs or intentions
regarding our business, technologies and products, financial condition,
strategies or prospects, including statements regarding our expectations
about Rayaldy™, its market potential, and that it will address
the approximately 4 million CKD stage 3 and 4 patients in the U.S. and
many more elsewhere, with SHPT and vitamin D insufficiency, that we will
be able to successfully develop, obtain approval for and launch sales of
Rayaldy™, the expected completion dates for our trials and that top-line
data will be available in mid-2014. Many factors could cause our
actual activities or results to differ materially from the activities
and results anticipated in forward-looking statements. These factors
include those described in our filings with the Securities and Exchange
Commission, as well as risks inherent in funding, developing and
obtaining regulatory approvals of new, commercially-viable and
competitive products and treatments, including the risks that the Phase
3 clinical trials for Rayaldy™ may not be successful or achieve the
expected results or effectiveness, and may not generate data that would
support the approval or marketing of this product for the indications
being studied, that others may develop products which are superior to
Rayaldy TM , and that Rayaldy TM may
not have advantages or prove to be superior over presently marketed
products, including the currently used high monthly doses of
prescription vitamin D 2, activated vitamin D
hormone and over-the-counter vitamin D supplements . In addition,
forward-looking statements may also be adversely affected by general
market factors, competitive product development, product availability,
federal and state regulations and legislation, the regulatory process
for new products and indications, manufacturing issues that may arise,
patent positions and litigation, among other factors. The
forward-looking statements contained in this press release speak only as
of the date the statements were made and we do not undertake any
obligation to update forward-looking statements. We intend that all
forward-looking statements be subject to the safe-harbor provisions of
the PSLRA.
OPKO Health, Inc.
Steven D. Rubin or Juan F. Rodriguez, 305-575-4100
Source: OPKO Health, Inc.
Released December 19, 2013
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